Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.
Gene Therapy & Mesothelioma
Posted on: 18 June 2017, source: mesothelioma.com
Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time that’s passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.
Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time that’s passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.
Altered virus may expand patient recruitment in human gene therapy trials
Posted on: 14 June 2017, source: Science Daily
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.
Allergy Breakthrough with Gene Therapy
Posted on: 8 June 2017, source: World Health
It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.
It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.
European Child Receives Strimvelis Gene-Therapy Treatment
Posted on: 3 May 2017, source: Rare Disease Report
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.
uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe
Posted on: 20 April 2017, source: UniQure
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017. "The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017. "The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."
Interview with Daniel Leonard, Director, Global Patient Advocacy, UniQure
Posted on: 11 April 2017, source: Gene Therapy for Rare Disorders 2017
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.
AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
Posted on: 23 March 2017, source: press release
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.
Gene Therapy News
- Gene therapy upstart lands $45M round; Biogen picks new communications chief - Endpoints News
Wed, 28 Jun 2017 21:43 GMT - Gene Therapy Vectors Come to Grips with Nervous System, Root and Branch - Genetic Engineering & Biotechnology News
Wed, 28 Jun 2017 14:31 GMT - Gene therapy co LogicBio raises $50m - Globes
Wed, 28 Jun 2017 13:37 GMT - Cancer Gene Therapy Market by way of study, synthesis and summation 2017 - Equity Insider (press release)
Wed, 28 Jun 2017 13:27 GMT - Hammered on a gene therapy setback, Dimension cuts staff and circles the wagons - Endpoints News
Wed, 28 Jun 2017 13:20 GMT
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Gene Therapy Literature
Scroll through the latest articles in gene therapy scientific journals.
Recent early view article in Journal of Gene Medicine:
Recent early view article in Journal of Gene Medicine: